On May 6, 2016 Oncolytics Biotech Inc. (TSX: ONC) (OTCQX: ONCYF) (FRA: ONY) ("Oncolytics" or the "Company") reported its financial results and operational highlights for the first quarter ended March 31, 2016 (Filing, Q1, Oncolytics Biotech, 2016, MAY 6, 2016, View Source [SID:1234512055]).

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"We recently reported data from two sponsored, randomized Phase 2 studies, highlighting reduced tumor burden in ovarian cancer and improved longer term survival in pancreatic cancer," said Dr. Brad Thompson. "The data from these studies will be very useful in identifying potential indications and study designs that we could advance into later stage studies in the future."

Selected Highlights

Since January 1, 2016, selected highlights announced by the Company include:

Clinical Program

· Treatment of the first patients in a Phase Ib study of pembrolizumab (KEYTRUDA) in combination with REOLYSIN and chemotherapy in patients with advanced pancreatic adenocarcinoma, the Company’s first trial examining REOLYSIN in combination with a checkpoint inhibitor;
· Updated results from a randomized Phase 2 clinical trial of its lead product, REOLYSIN, in combination with paclitaxel in patients with ovarian cancer (GOG-0186H), where an intent-to-treat analysis, as assessed by CA-125 antigen levels, showed statistically significantly reduction in tumor burden;
· Updated results from a randomized Phase 2 clinical trial of its lead product, REOLYSIN, in combination with carboplatin and paclitaxel in patients with pancreatic cancer (NCI-8601), where an intent-to-treat analysis of overall survival on patients with confirmed treatment regimes, as assessed by the percentage of patients surviving for two years, showed a statistically significantly higher percentage of patients surviving two years in the test arm versus the control arm (p = 0.001), the crossover arm versus the control arm (p = 0.03) and the test plus crossover arms versus the control arm (p = 0.0004);
Basic Research

· Two poster presentations covering preclinical work in multiple myeloma and colorectal cancer being made by the Company’s research collaborators at the 2016 American Association of Cancer Research annual meeting;
Financial

· Entry into an "at-the-market" equity distribution agreement with Canaccord Genuity Corp. permitting Oncolytics at its sole discretion, from time to time and until March 16, 2018, to sell common shares having an aggregate offering value of up to $4.6 million; and
· At March 31, 2016 the Company reported $22.3 million in cash, cash equivalents and short-term investments. At May 5, 2016, the Company had approximately $21.4 million in cash, cash equivalents and short-term investments, which is expected to provide sufficient funds to support several small early-stage immunotherapy combination studies as well as both a run-in and a registration study in muscle-invasive bladder cancer.

ONCOLYTICS BIOTECH INC.
INTERM CONSOLIDATED STATEMENTS OF FINANCIAL POSITION

As at March 31,
2016
$ December 31,
2015
$
Assets
Current assets
Cash and cash equivalents 20,233,408 24,016,275
Short-term investments 2,088,800 2,060,977
Accounts receivable 59,648 340,059
Prepaid expenses 229,288 506,669
Total current assets 22,611,144 26,923,980

Non-current assets
Property and equipment 411,762 459,818
Total non-current assets 411,762 459,818

Total assets 23,022,906 27,383,798

Liabilities And Shareholders’ Equity
Current Liabilities
Accounts payable and accrued liabilities 2,554,338 2,709,492
Total current liabilities 2,554,338 2,709,492

Shareholders’ equity
Share capital
Authorized: unlimited
Issued:
March 31, 2016 – 118,697,122
December 31, 2015 – 118,151,622 261,224,148 261,324,692
Contributed surplus 26,359,606 26,277,966
Accumulated other comprehensive income 590,919 760,978
Accumulated deficit (267,706,105) (263,689,330)
Total shareholders’ equity 20,468,568 24,674,306
Total liabilities and equity 23,022,906 27,383,798

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF LOSS AND COMPREHENSIVE LOSS

2016 2015
For the three month period ending March 31 $ $
Expenses
Research and development 2,726,129 2,425,539
Operating 1,360,412 1,182,734
Operating loss (4,086,541) (3,608,273)
Interest 69,621 56,435
Loss before income taxes (4,016,920) (3,551,838)
Income tax expense 145 —
Net loss (4,016,775) (3,551,838)
Other comprehensive income items that may be
reclassified to net loss
Translation adjustment (170,059) 225,591

Net comprehensive loss (4,186,834) (3,326,247)
Basic and diluted loss per common share (0.03) (0.04)

Weighted average number of shares (basic and diluted) 118,199,985 99,557,654

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF CHANGES IN EQUITY


Share Capital
$

Contributed
Surplus
$

Accumulated
Other
Comprehensive
Income
$

Accumulated
Deficit
$

Total
$

As at December 31, 2014 237,657,056 25,848,429 280,043 (249,966,335) 13,819,193

Net loss and comprehensive income — — 225,591 (3,551,838) (3,326,247)
Issued, pursuant to Share Purchase
Agreement 1,925,596 — — — 1,925,596
Issued, pursuant to "At the Market"
Agreement 14,636,918 — — — 14,636,918
Share based compensation — 114,970 — — 114,970
As at March 31, 2015 254,219,570 25,963,399 505,634 (253,518,173) 27,170,430

Share Capital
$

Contributed
Surplus
$

Accumulated
Other
Comprehensive
Income
$

Accumulated
Deficit
$

Total
$

As at December 31, 2015 261,324,692 26,277,966 760,978 (263,689,330) 24,674,306

Net loss and comprehensive income — — (170,059) (4,016,775) (4,186,834)
Issued, pursuant to Share Purchase
Agreement
— — — — —
Issued, pursuant to "At the Market"
Agreement (100,544) — — — (100,544)
Share based compensation — 81,640 — — 81,640
As at March 31, 2016 261,224,148 26,359,606 590,919 (267,706,105) 20,468,568

ONCOLYTICS BIOTECH INC.
INTERIM CONSOLIDATED STATEMENTS OF CASH FLOWS

2016 2015
For the three month period ending March 31 $ $

Operating Activities
Net loss for the period (4,016,775) (3,551,838)
Amortization – property and equipment 45,942 45,130
Share based compensation 81,640 114,970
Impact of unrealized foreign exchange (gains) losses 141,295 (305,156)
Net change in non-cash working capital 724,655 949,705
Cash used in operating activities (3,023,243) (2,747,189)

Investing Activities
Acquisition of property and equipment — (11,940)
Purchase of short-term investments (27,823) (29,292)
Cash used in investing activities (27,823) (41,232)

Financing Activities
Proceeds from Share Purchase Agreement — 1,925,596
Proceeds from "At the Market" equity distribution agreement (100,544) 14,636,918
Cash provided by financing activities (100,544) 16,562,514
(Decrease) increase in cash (3,151,610) 13,774,093
Cash and cash equivalents, beginning of period 24,016,275 14,152,825
Impact of foreign exchange on cash and cash equivalents (631,257) 651,105
Cash and cash equivalents, end of period 20,233,408 28,578,023

To view the Company’s Fiscal 2016 First Quarter Consolidated Financial Statements, related Notes to the Consolidated Financial Statements, and Management’s Discussion and Analysis, please see the Company’s quarterly filings, which will be available under the Company’s profile at www.sedar.com and on Oncolytics’ website at View Source

On May 6, 2016 ReCyte Therapeutics, a subsidiary of BioTime, Inc., a clinical-stage regenerative medicine company with a focus on pluripotent stem cell technology, reported the publication of a scientific paper titled, "A novel lineage restricted, pericyte-like cell line isolated from human embryonic stem cells," by Midori Greenwood-Goodwin, Jiwei Yang, Mohammad Hassanipour, and David Larocca (Press release, BioTime, MAY 6, 2016, View Source;p=RssLanding&cat=news&id=2166093 [SID:1234512020]). The article was released in the peer-reviewed online publication in Scientific Reports. The online version of the article can be found at www.nature.com/articles/srep24403.

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In the article, researchers at ReCyte Therapeutics demonstrate the successful isolation of a new type of human blood vessel support cell that is involved in the formation and stabilization of blood vessels, a process known as angiogenesis. Unlike typical angiogenic support cells which are multi-talented and can turn into other types of tissues such as bone, these precursor cells, termed PC-M cells, are highly specialized to support and strengthen blood vessels.

"When you want a specific job done, its best to call a specialist for treatment," said Dr. David Larocca, Vice President of Research and Development. "That’s why we believe these novel pericyte-like cells may be an ideal source of cells for promoting new blood flow in patients suffering from ischemic vascular disease (IVD) where clogged arteries have blocked blood flow to limbs or the heart."

Left unchecked, IVD often leads to amputation or heart failure for patients that have failed, or are not candidates for surgical interventions. Thus, there is a critical unmet medical need for new angiogenic therapies. Importantly, PC-M cells are highly stable and have a high capacity to replicate under standard laboratory conditions. They therefore can be manufactured on an industrial scale needed to treat a large patient population. At ReCyte, we are currently investigating the use of PC-M cells for treating critical limb ischemia, a late stage of IVD that afflicts diabetics and the elderly.

We have also developed a PC-M cell based co-culture assay kit called VascuNet (sold by Ascendance Biotechnology, Inc.) that’s designed to be used to screen for drugs that affect human blood vessel growth and stability. This novel assay mimics the body’s own angiogenic mechanisms for growing blood vessels which involves both supporting pericytes, and the endothelial cells that line the vessel walls. This biomimetic VascuNet assay can be used to discover new anti-angiogenic cancer drugs that kill tumors by cutting off or destabilizing their blood supply. It can also be used to screen for pro-angiogenic drugs that increase blood flow for treating ischemia.

The paper, based off of BioTime/ReCyte’s extensive research and discoveries involving iPS cells, if developed, could potentially bring value to the pharmaceutical industry interested in developing cardiovascular and cancer therapies. This development ties into BioTime’s broader strategy to allow its non-core therapeutic assets to mature into successful regenerative cell therapies, for a wide variety of degenerative diseases, to help address the critical unmet medical needs of patients worldwide.

On May 06, 2016 CytomX Therapeutics, Inc. (Nasdaq:CTMX), a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, reported financial results for the first quarter ending March 31, 2016 (Press release, CytomX Therapeutics, MAY 6, 2016, View Source;p=RssLanding&cat=news&id=2165975 [SID:1234511991]).

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"Since our IPO, we have maintained strong strategic and operational momentum as demonstrated by our partnership with AbbVie and the advancement of our wholly-owned programs towards the clinic," said Sean McCarthy, D.Phil., president and chief executive officer of CytomX Therapeutics. "With this progress and our strong cash position we are well-positioned to build a multi-product company to transform antibody therapeutics for the treatment of cancer."

As of March 31, 2016, CytomX had cash and cash equivalents and short-term investments of $180.6 million. The Company has updated its financial guidance and expects net cash utilization of $20.0 to $25.0 million in 2016, reduced from its previous guidance of $45.0 to $50.0 million. The Company is maintaining its guidance that its existing capital resources will be sufficient to fund its anticipated operations through 2018.

Business Highlights and Recent Developments

On April 21, 2016, the Company announced that it had entered into a collaboration with AbbVie to co-develop and co-commercialize Probody Drug Conjugates against CD71, with CytomX leading pre-clinical and early clinical development. AbbVie will lead later development and commercialization, with global late-stage development costs shared between the two companies. CytomX received an upfront payment of $30 million and is eligible to receive up to $470 million in development, regulatory and commercial milestones, pending the achievement of pre-determined outcomes. AbbVie will lead global commercial activities with CytomX eligible to receive a profit share in the U.S. and tiered double-digit royalties on net product sales outside of the U.S. CytomX retains an option to co-promote in the U.S.

On April 19, 2016, the Company presented data from its PD-1 and CD71 programs at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting evidencing efficacy and improved tolerability for Probody therapeutics in preclinical models.

The Company remains on track to file an IND for CX-072, a Probody therapeutic targeting PD-L1, in the second half of 2016 and an IND for CX-2009, a Probody drug conjugate targeting CD166, in the first half of 2017.

During the first quarter, the Company announced selection of a third target by Bristol-Myers Squibb in accordance with the companies’ strategic oncology collaboration established in May 2014, triggering a $10 million milestone payment.

First Quarter Financial Results

Cash, cash equivalents and investments totaled $180.6 million as of March 31, 2016, compared to $186.7 million as of December 31, 2015. The decrease reflects cash used in operations offset by a $10.0 million milestone payment received from Bristol-Myers Squibb in connection with their third target selection in January 2016.

Research and development expenses were $13.4 million for the first quarter of 2016, compared to $4.7 million for the first quarter of 2015. The increase was primarily attributable to $4.9 million in manufacturing costs for the Company’s CX-072 and CX-2009 programs in preparation for preclinical and clinical studies, $1.3 million in laboratory and professional services, $1.3 million in non-cash stock based compensation due to higher stock valuation, $0.8 million in personnel-related expenses due to an increase in headcount and $0.4 million in royalty payments to a third party triggered by Bristol-Myers Squibb’s milestone payment in connection with its third target selection. The Company expects the manufacturing costs for the two programs to continue into the second quarter and the costs related to preparation for CX-072 clinical trials to increase in the third quarter.

General and administrative expenses were $5.0 million for the first quarter of 2016, compared to $1.9 million for the first quarter of 2015. The increase was primarily attributable to $1.2 million in additional consulting and professional service expenses associated with operating as a public company, $1.1 million in non-cash stock based compensation due to higher stock valuation and $0.7 million in personnel-related expenses due to an increase in headcount.

On May 6, 2016 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") that its European regional headquarters Eisai Europe Ltd. (Location: U.K.) has received from the European Commission approval for anticancer agent Halaven (eribulin mesylate) for the treatment of adult patients with unresectable liposarcomas who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease (Press release, Eisai, MAY 6, 2016, View Source [SID:1234511992]). Halaven is the first and only single agent to demonstrate a statistically significant overall survival (OS) benefit in a Phase III trial in patients with advanced, recurrent or metastatic soft tissue sarcoma (liposarcoma or leiomyosarcoma). Following approval for use in the treatment of metastatic breast cancer, this marks the second indication for which Halaven has received approval based on an extension of OS.

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The approval was based on the results of a Phase III study (Study 309)1 comparing the efficacy and safety of Halaven versus dacarbazine in 452 patients (aged 18 or over) with locally advanced, recurrent or metastatic soft tissue sarcoma (liposarcoma or leiomyosarcoma) who had disease progression following standard therapies which must have included an anthracycline and at least one other additional regimen. Halaven demonstrated a statistically significant extension in the study’s primary endpoint of OS over the comparator treatment dacarbazine (Halaven median OS: 13.5 months vs dacarbazine median OS: 11.5 months; Hazard Ratio (HR) 0.77 [95% CI=0.62-0.95], p=0.0169). For patients with liposarcoma, Halaven demonstrated a significant improvement in OS over dacarbazine (Halaven, median OS: 15.6 months vs dacarbazine, median OS: 8.4 months; HR 0.51 [95% CI=0.35-0.75]).
In this study, the most common treatment-emergent adverse events (incidence greater than or equal to 25%) in patients treated with Halaven were fatigue, neutropenia, nausea, alopecia, constipation, peripheral neuropathy, abdominal pain, and pyrexia, which was consistent with the known side-effect profile of Halaven.

Halaven is a halichondrin class microtubule dynamics inhibitor with a distinct binding profile. Recent non-clinical studies showed that Halaven is associated with increased vascular perfusion and permeability in tumor cores.2 Halaven promotes the epithelial state and decreases the capacity of breast cancer cells to migrate. 3 Halaven is currently approved for use in the treatment of breast cancer in approximately 60 countries including Japan and countries in Europe, the Americas and Asia. Halaven was approved in the United States for the treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracycline-containing regimen in January 2016, and was approved in Japan for the treatment of soft tissue sarcoma in February 2016.
Soft tissue sarcoma is a collective term for a diverse group of malignant tumors that occur throughout the soft tissue (fat, muscle, nerves, fibrous tissues and blood vessels). Approximately 29,000 patients in Europe are diagnosed with soft tissue sarcoma each year or about 1% of all cancers diagnosed in Europe. Liposarcoma is one of the most common forms of soft tissue sarcoma. As outcomes are poor for patients with advanced disease, it remains a disease with significant unmet medical need. Eisai Co., Ltd. No.16-29 May 6, 2016 Eisai remains committed to providing additional clinical evidence for Halaven aimed at maximizing value of the drug as it seeks to contribute further to addressing the diverse needs of, and increasing the benefits provided to, patients with cancer, their families, and healthcare providers.

About Halaven
Halaven is the first in the halichondrin class of microtubule dynamics inhibitors with a novel mechanism of action. Structurally Halaven is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai. Halaven is believed to work by inhibiting the growth phase of microtubule dynamics which prevents cell division. In addition, recent non-clinical studies showed that Halaven is associated with increased vascular perfusion and permeability in tumor cores.2 Halaven promotes the epithelial state and decreases the capacity of breast cancer cells to migrate.3

Halaven was first approved in November 2010 in the United States as a treatment for patients with metastatic breast cancer who have received at least two chemotherapeutic regimens for the treatment of metastatic disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting. Halaven is currently approved for use in the treatment of breast cancer in approximately 60 countries worldwide, including Japan and countries in Europe, the Americas and Asia. In Japan, Halaven has been approved to treat inoperable or recurrent breast cancer and was launched in the country in July 2011. In addition, Halaven has been approved in countries in Europe and Asia indicated as a treatment for patients with locally advanced or metastatic breast cancer who have progressed after at least one chemotherapeutic regimen for advanced disease. Prior therapy should have included an anthracycline and a taxane in either the adjuvant or metastatic setting, unless patients were not suitable for these treatments.

Regarding soft tissue sarcoma, Halaven was approved in the United States for the treatment of patients with unresectable or metastatic liposarcoma who have received a prior anthracycline-containing regimen in January 2016, approved in Japan for the treatment of soft tissue sarcoma in February 2016, and approved in Europe for the treatment of adult patients with unresectable liposarcomas who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease in May 2016. Applications seeking approval for use in the treatment of soft tissue sarcoma are currently under review in Switzerland, Russia, Australia, Brazil, and Malaysia. Furthermore, Halaven has been designated as an orphan drug for soft-tissue sarcoma in the United States and Japan.

2. About Study 3091
Conducted primarily in Europe and the United States, Study 309 was a multicenter, open-label, randomized Phase III study comparing the efficacy and safety of Halaven versus dacarbazine in 452 patients (aged 18 or over) with locally advanced, recurrent or metastatic soft tissue sarcoma (liposarcoma or leiomyosarcoma) who had disease progression following standard therapies which must have included an anthracycline and at least one other additional regimen. Patients received either Halaven (1.4 mg/m2 administered intravenously on Day 1 and Day 8) or dacarbazine (850–1200 mg/m2 administered intravenously on Day 1) every 21 days until disease progression.

From the results for the study, Halaven demonstrated a statistically significant extension in the study’s primary endpoint of overall survival (OS) over the comparator treatment dacarbazine (Halaven median OS: 13.5 months vs dacarbazine median OS: 11.5 months; Hazard Ratio (HR) 0.77 [95% CI=0.62-0.95], p=0.0169). Furthermore, in the study’s secondary endpoints, there was no statistically significant difference found between Halaven and dacarbazine in either progression-free survival (PFS) (median PFS: 2.6 months in both arms) or progression-free rate at 12 weeks (PFR12wks) (Halaven PFR12wks: 33% vs dacarbazine PFR12wks: 29%).

For patients with liposarcoma (143 patients), Halaven demonstrated a statistically significant improvement in OS over dacarbazine (Halaven, median OS: 15.6 months vs dacarbazine, median OS: 8.4 months; HR 0.51 [95% CI=0.35-0.75]).

In this study, the most common treatment-emergent adverse events (incidence greater than or equal to 25%) in patients treated with Halaven were fatigue, neutropenia, nausea, alopecia, constipation, peripheral neuropathy, abdominal pain, and pyrexia, which was consistent with the known side-effect profile of Halaven.

3. About Soft Tissue Sarcoma
Soft tissue sarcoma is a collective term for a diverse group of malignant tumors that occur throughout the soft tissue (including fat, muscle, nerves, fibrous tissues and blood vessels). Approximately 12,000 patients in the United States and 29,000 patients in Europe are diagnosed with soft tissue sarcoma each year. According to a patient survey conducted by Japan’s Ministry of Health, Labour and Welfare, there are approximately 4,000 patients with soft tissue sarcoma in Japan. As the structures where the tumors originate are diverse, there are various types of soft tissue sarcoma, and the most common types include leiomyosarcoma, liposarcoma and malignant fibrous histiocytoma. While treatment of soft tissue sarcoma is focused on curative surgery, if the stage of the disease is advanced, treatment then becomes a combination of chemotherapy and radiation therapy. As outcomes are poor for patients with advanced disease, it remains a disease with significant unmet medical need.

On May 5, 2016 Mustang Bio, Inc. ("Mustang"), a Fortress Biotech (NASDAQ: FBIO) Company, reported that an oral presentation related to its MB-101 (IL13Rα2-specific CAR T cells) product candidate in development was presented by City of Hope ("COH") Investigators at the American Society of Gene and Cell Therapy 19th Annual Meeting (ASGCT) (Free ASGCT Whitepaper) at the Marriott Wardman Park Hotel in Washington, DC (Filing, 8-K, Fortress Biotech, 2016, MAY 6, 2016, View Source [SID:1234512023]).

Michael S. Weiss, Mustang Bio’s Executive Chairman, Interim Chief Executive Officer and President commented on the data, "We are very encouraged by the early safety and efficacy profile demonstrated by MB-101 (IL13Rα2-specific CAR T cells) to date. We were particularly impressed with the data presented today showing a patient treated at the first dose level who obtained an investigator designated complete response to MB-101. We believe this is the first demonstration of a response in a GBM patient utilizing CAR-T treatment. We are eager to continue the dose escalation to further assess the safety, activity and durability of MB-101 treatment at higher doses." Mr. Weiss continued, "We would like to thank the investigators at City of Hope for all of their efforts on this important research program."

The following summarizes the oral presentation today:

Phase I Study of Second Generation Chimeric Antigen Receptor–Engineered T cells Targeting IL13Rα2 for the Treatment of Glioblastoma

The Phase I study abstract that was presented showed early data evaluating the safety, feasibility and bioactivity of weekly intracranial infusions of autologous IL13Rα2-specific CAR T cells in patients with recurrent IL13Rα2+ GBM. On this study, patients are treated on a 4-week therapeutic regimen consisting of 3 weekly intracranial infusions of IL13Rα2-specific CAR T cells followed by one rest week for toxicity and disease assessment. To date, treatment of the first low dose cohort of patients has been completed demonstrating that local delivery of IL13Rα2-specific CAR T cells post-surgical resection appeared to be safe and well-tolerated, with no grade 3 or higher toxicities attributed to the therapy reported. Importantly, early evidence for antitumor activity following CAR T cell administration was observed.

One patient of particular interest presented with a recurrent multifocal GBM, including one metastatic site in the spine and extensive leptomeningeal disease. This patient was initially treated per protocol with six local infusions of IL13Rα2-specific CAR T cells into the resection cavity of the largest recurrent tumor focus in the posterior temporal-occipital region. Encouragingly, this CAR T cell injection site remained stable without evidence of disease recurrence for over 7-weeks, while other disease foci distant from the CAR T cell injection site continued to progress. This patient was then treated on a compassionate use protocol with five weekly intraventricular infusions of IL13Rα2-specific CAR T cells without any other therapeutic interventions. The investigator reported today that following treatment the patient achieved a complete response. Early clinical findings suggest that intracranial delivery of second-generation IL13Rα2-targeted CAR T cells is safe and well-tolerated, and that after adoptive transfer, CAR T cells survive and maintain activity, capable of eliciting potent antitumor responses against recurrent multifocal glioblastoma.

About Glioblastoma multiforme (GBM)
Glioblastomas (GBM) are tumors that arise from astrocytes cells that make up the supportive tissue of the brain. These tumors are usually highly malignant (cancerous) because the cells reproduce quickly and they are supported by a large network of blood vessels. GBM is the most common brain and central nervous system (CNS) malignancy, accounting for 15.1% of all primary brain tumors, and 55.1% of all gliomas. There are an estimated 12,120 new glioblastoma cases predicted in 2016 in the U.S. Malignant brain tumors are the most common cause of cancer-related deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19 year olds in the U.S. (Brain Tumor Statistics. American Brain Tumor Association. December 2015). While GBM is a rare disease (2-3 cases per 100,000 person life years in the U.S. and E.U.), it is quite lethal with 5-year survival rates historically less than 10%. Chemotherapy with temozolomide and radiation are shown to extend mean survival from ~12 to ~15 months, while surgery remains the standard of care. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies. Treatment is further complicated by the susceptibility of the brain to damage, difficulty of the brain to repair itself and limitation to drugs crossing the blood brain barrier. Immunotherapy approaches targeting brain tumors offer promise over conventional treatments.

About MB-101 (IL13Rα2-specific CAR-T cells)
IL13Rα2 is an attractive target for CAR-T therapy as it has limited expression in normal tissue but is over-expressed on the surface of the majority of GBM. CAR T cells designed to express a membrane-tethered IL-13 receptor ligand (IL-13) incorporating a single point mutation that provides high affinity for IL13Rα2 and reduces binding to IL13Rα1 in order to reduce healthy tissue targeting.

We are developing an optimized CAR-T product incorporating enhancements in CAR design and T cell engineering to improve antitumor potency and T cell persistence. We include a second generation hinge optimized CAR containing mutations in the IgG4 linker to reduce off-target Fc interactions, as well as the 41BB (CD137) co-stimulatory signaling domain for improved persistence of CAR T cells, and extracellular domain of CD19 as a selection/safety marker. In order to further improve persistence, central memory T cells are enriched and genetically engineered using a manufacturing process that limits ex vivo expansion in order to reduce T cell exhaustion and maintain a memory T cell phenotype.

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